Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase a cDNA to nonhuman primates

Human Gene Therapy Clinical Development

Volumn 25, Issue 3, 2014, Pages 164-177

  Rosenberg, Jonathan B ^a   Sondhi, Dolan ^a   Rubin, David G ^a   Monette, Sébastien ^b   Chen, Alvin ^a   Cram, Sara ^a   De, Bishnu P ^a   Kaminsky, Stephen M ^a   Sevin, Caroline ^c,d   Aubourg, Patrick ^c,d   Crystal, Ronald G ^a  

^a WEILL CORNELL MEDICAL COLLEGE   (United States)

^b MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^c INSERM   (France)

^d BICÊTRE HOSPITAL   (France)

Author keywords

[No Author keywords available]

Indexed keywords

CEREBROSIDE SULFATASE; COMPLEMENTARY DNA; MANNITOL; PHOSPHATE BUFFERED SALINE;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ARTICLE; CAUDATE NUCLEUS; CENTRAL NERVOUS SYSTEM; CHOROID PLEXUS; COMPARATIVE STUDY; CONTROLLED STUDY; DRUG ADMINISTRATION ROUTE; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG SAFETY; GENE EXPRESSION; GENE VECTOR; GENOME; GRAY MATTER; HISTOPATHOLOGY; HUMAN; IMMUNOHISTOCHEMISTRY; INTRAARTERIAL DRUG ADMINISTRATION; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; INTRAPARENCHYMAL DRUG ADMINISTRATION; LABORATORY; LATERAL BRAIN VENTRICLE; MIDDLE CEREBRAL ARTERY; NONHUMAN; PRIMATE; PUTAMEN; SEROTYPE; SPINAL CORD; SPINAL GANGLION; THALAMUS; WHITE MATTER; ANIMAL; DEFICIENCY; DEPENDOPARVOVIRUS; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER; GENETICS; LEUKODYSTROPHY, METACHROMATIC; METABOLISM; PATHOLOGY; PROCEDURES; TRANSGENE;

ANIMALS; CENTRAL NERVOUS SYSTEM; CEREBROSIDE-SULFATASE; DEPENDOVIRUS; DNA, COMPLEMENTARY; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LEUKODYSTROPHY, METACHROMATIC; PRIMATES; SEROGROUP; TRANSGENES;

EID: 84907276974     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2013.239     Document Type: Article

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